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Monday, December 16, 2013

An interview with GlaxoSmithKline's Dr. Jan Thirkettle


An interview with:

Jan Thirkettle, BSc, PhD
Head, Advanced Therapy Delivery
GlaxoSmithKline Medicines Research Centre

In anticipation of Phacilitate's 2014 Cell and Gene Therapy Forum to be held at the end of January in Washington, DC, I invited the Phacilitate team to provide blog readers with an interview of one of their upcoming faculty. The result is the very interesting Q&A thread you see below about GSK's call and gene therapy program combined with some of his thoughts on the sector.  

Phacilitate: You head the Advanced Therapy Delivery group at GSK – please could you outline your specific goals and activities as an organisation for us? Which projects/technologies are you prioritising currently, and why?

Dr. Thirkettle: Our immediate priority is to deliver the Chemistry, Manufacturing and Controls (CMC) aspects of a collaboration we already have for an investigational ex vivo gene therapy currently in late stage development with TIGET, the San Raffaele Institute for Gene Therapy in Milan. 

Through this collaboration we believe that we have a responsibility to complete the process for development of gene therapy, get it to file and make it available to more patients. We are really excited by the clinical data which TIGET have generated on the current programs thus far, but this is also laying really important groundwork for how we will work on other products in that collaboration and giving us real insight into the issues which we need to tackle in order to be able to progress following opportunities in this collaboration and make these autologous therapies more scalable.

This feeds into our second priority, which is to develop more efficient manufacturing technologies that can allow us to reduce the cost of goods, reduce the complexity, and increase the scalability of ex vivo gene therapy products. This is important within our existing collaboration but also it is going to be critical to allow us to engage with other opportunities which might have larger patient numbers.

Thirdly, whilst the immediate focus of our group is on the CMC aspects (process development, vectorology, cell characterisation, QC), we are also very focussed on working across the GSK platform organisation to leverage the expertise we have from our pharma and vaccine background and which is relevant to this area. This engagement and strategic planning is also critical to make sure that we build expertise in all of the other really key functions; for instance, this is an area where really strong understanding of the regulatory and the quality aspects is absolutely critical.

Phacilitate: This January, you will be joining a panel of big pharma representatives tasked with answering the questions of whether 2014 is the year pharma get serious about Cell & Gene Therapy, and whether it has been a lack of innovation or a lack of proven products that has held them back in the past - what’s your initial response to these questions?

Dr Thirkettle: I’m not sure it’s fair to say that big pharma has yet to get serious, if you look at the investments and deals of the past 3 years. I certainly don’t see any lack of innovation out there, it is almost the opposite. We talk about cell and gene therapy as a modality, but it is a huge, broad field; as broad as ‘biopharmaceuticals’ which is a field with many approaches and technologies. 

Within the cell and gene therapy field we have a very large number of modalities, different cell types, different approaches, and a huge diversity of technologies. And they are all evolving at a really blistering pace. So one of the challenges for anybody who is outside of the groups who are really driving that core science is to know which is the right bit to grab hold of and it’s not the sort of area that you can just dabble in if you want to be really be effective and add value.

Rather than lack of innovation, then, the challenge is maybe more of knowing how to pick the winners at the right time and understand the potential utility of that technology/product at this early stage. I think the key thing is that it requires a number of elements to come together; you need the biology and the underpinning science to be strong; you need a therapy with good clinical data to really evidence that and discharge some of the risk; and then you need an understanding of how broadly applicable that technology might be - is there some sort of platform that sits behind it that spreads your risk? And finally, you need an understanding if whether you can actually make that product, can you scale it up?

Phacilitate: You are also kindly chairing a session which explores the long-standing but still critical challenges in developing cost effective and robust manufacturing models for cell & gene therapies at industrial scale. What progress do you see being made in this regard? To what extent will it be a case of these problems being solved relatively quickly and easily as big pharma come on board more and more, as some have suggested?

Dr Thirkettle: There are some massive challenges, but I suppose I would never underestimate the ability of really committed scientists, whether from academia or biotechs or big pharma, to solve those problems given enough focus and time.
I think the key is creating a focus because in a way it goes back to the previous point - this is a hugely broad field. When you stand back and look at this mass of really exciting science and opportunity, the challenge actually is to understand where to focus that investment in time and money. It becomes an awful lot easier to create that focus and energy to invest where you have got really compelling clinical data - you know you have got something that can potentially be a product. Equally if you can have confidence that a technology has multi product utility, your ability to invest is that much greater. If you can make a strong case on either of those points then you’re in a good position to create the focus needed to drive investment in the manufacturing and commericalisation aspects.

Phacilitate: Finally, what are you hoping to get out your participation in the meeting this January?

Dr Thirkettle: I’m really hoping first of all to meet lots of other people who are facing the same challenges and learning the same things. I think it is really exciting to see the journey over the past few year with increasing discussions around “How do we make these therapies? ” rather than “Will these therapies ever work?” This is a field which would potentially be greatly enabled if there players could find a way to share learnings as much as possible about what works and what doesn’t. There is a huge opportunity to work in a pre-competitive mode. It is in everybody’s interest because there are so many challenges, so many things to learn. I think this is a forum which makes it really easy to have those conversations, and start to build those relationships that, ultimately, should help everyone.

Friday, June 28, 2013

Cell Therapy Industry Infographic


Here is an infographic of the data in one of my recent presentations.  

Enjoy. Consume. Share. Critique.

Sunday, April 21, 2013

Commercialization of Regenerative Medicine: Learning from Spin-Outs


The meeting “Commercialization of Your Regenerative Medicine Research: Lessons from Spin Out Successes” was hosted by the Oxbridge Biotech Roundtable (OBR) (Oxford, UK) at the University of Oxford in February, 2013, and attracted a multi-stakeholder audience spanning academia and industry. 

The event featured case studies from Gregg Sando, CEO, Cell Medica (London, UK), John Sinden, CSO, Reneuron (Guilford, UK), and Paul Kemp, CEO and CSO, Intercytex (Manchester, UK). 

OBR is a student-led initiative with over 7000 members across eight different UK and US locations with a mission to foster a conversation about the healthcare and life sciences industry. 

Anna French and David A. Brindley, along with some of my assistance, captured and have now published the main themes of the meeting and the major questions facing the regenerative medicine industry and its rapidly emerging subsets of cellular and gene therapies. 

Notably, we discuss the compatibility of regenerative therapies to the existing healthcare infrastructure, biomanufacturing challenges (including scalability and comparability), and the amenability of regenerative therapies to existing reimbursement and investment models. Furthermore, we reiterate key words of advice from seasoned industry leaders intended to accelerate the translation path from lab bench to the marketplace.

To read the review see: Commercialization of Regenerative Medicine: Learning from Spin-Outs

Anna French, R. Lee Buckler, and David A. Brindley. Rejuvenation Research. April 2013, 16(2): 164-170. doi:10.1089/rej.2013.1423.

Wednesday, April 17, 2013

2013 Annual Regenerative Medicine Industry Report


The Alliance for Regenerative Medicine announced today the release of the 2013 annual regenerative medicine industry report.  Here is the announcement in the Wall Street Journal online.

I'm proud to have been a part of putting it together and hope people find it useful.  It is available for download on the ARM website here.  

In addition to the complete download, ARM will make many of the figures, charts,  tables and sections available for members to download and use in their own publications and presentations. Watch for these resources to be announced soon.

Monday, January 21, 2013

2012 Financing Recap: Cell Therapy and Cell-Based Regenerative Medicine Companies


In October we posted a teaser saying that we had at that time tracked the infusion of over $1 billion dollars into cell therapy and cell-based regenerative medicine companies in 2012.  We promised details.  Here they are:

For the first half of the year, see our post "Monthly recap of funds raised by cell therapy & regenerative medicine companies" dated July 31 and updated August 8.

Since that post, we back-filled the following deals which we missed earlier:

  • In February, Stemgent raised $6.3M from eight private investors
  • In April, BioRestorative Therapies raised $2.1M from undisclosed private parties
  • In July, Immusoft received a $300,000 grant from NIH-SITR.
August saw the following financings:

  • Cell Cure Neurosciences was awarded $1.3M from Israel's Office of the Chief Scientist
  • Stemgent collected another $11.3M from eight private backers
  • Neuralstem brought in $2.76M in a public offering
  • Fate Therapeutics received $9.2M from undisclosed investors
September was a busy month with the following deals and grants:
  • Cellerant benefits from a further BARDA grant tranche with the potential to hit upwards of $36.4M
  • StemCells Inc saw the second of its CIRM-funded boats come in to the tune of $20M
  • Argos Therapeutics collected $16M from 24 different unnamed investors
  • Neuralstem succeeded in bringing in another $7M in a direct public offering
  • Pluristem rode its well-orchestrated fame to a $34M underwritten public offering
  • Advanced Cell Technology brought in $35M via ATM
  • Cellerant collected $2.09M from private investors
  • Cytori was awarded a BARDA grant with potential to hit up to $106M
  • Vital Therapies closed the first $16M from a multistage commitment to invest $76M by existing private investors. The investment is intended to fund three  Phase III trials of the company’s bioartificial liver therapy, ELAD, for two types of acute liver failure. One trial in the US (not yet recruiting) in acute alcoholic hepatitis involving 200 patients; the other two trial in EU and Australia/New Zealand for acute alcoholic hepatitis patients who fail steroid therapy and in subjects with fulminant hepatic failure
  • Avita Media rounded out the month with a modest $880,000 grant from AFIRM
So after seeing $172M come into the sector in Quarter One and $252M in Quarter Two, the Third Quarter saw companies in the sector glean commitments of up to $408M from various sources. 

The Fourth Quarter saw $288M infused into the sector broken down as follows:

  • Capricor gleaned a $19.8M from CIRM
  • China Cord Blood Corp secrured $50M in a convertible note financing from Golden Meditech Holding Limited
  • Cellerant kept the grant machine rolling with a $1.7M phase 1 SBIR grant from NCI
  • Tengion secured $15M in senior secured convertible notes from Celgene Corporation, RA Capital Management,  Deerfield Management Company, Bay City Capital and HealthCap
  • Viacyte saw a $10M grant from CIRM
  • Massachusetts-based Bluebird Bio finagled $9.36M from California's CIRM
  • Fibrocell Sciences did a $45M PIPE
  • Avita Medical gleaned $10M from a secondary public offering
  • Athersys closed a $23M secondary offering priced at $1.00
  • Tissue Regeneration Therapeutics eked out a $100,000 grant from Ontario Genomics Intitute
  • Quy Biosciences (formerly MedCell) raised $2.7M in a private placement
  • Kiadis Pharma managed a $12.8M financing round lead by LSP (Life Sciences Partners) and supported by a large investment from DFJ-Esprit. Other investors included Alta Partners, Quest for Growth and NOM.  The round will be used to do a late-stage phase 2 trial which they hope will position them for a solid pivotal with partner, Hospira.
  • NeoStem received a $1.2M grant from NIH
  • French biotech, TxCell raised €12.4M from existing shareholders to support an internationa ph 2b trial of Ovasave for the treatment of Crohn’s disease in patients who are refractory to current treatments.
  • In a Biotech Showcase presentation, Adaptimmune COO reported they were given 9.6M British Pounds by 'high-net-worth investors' in November to move forward their clinical pipeline
  • Northwest Biotherapeutics brought in $13.8M in a secondary public offering
  • Organovo secured $7.7M in a tender offer to warrant holders
  • Cytori closed a $23M secondary public offering (including the shoe)

Unfortunately neither Cell Therapy Group, nor any of our industry organizations, have any comprehensive financing data from year's past to know for certain whether broaching this $1,000,000,000 threshold is indeed a first-ever accomplishment.  We tracked $390M of investment into the sector in 2008 and then did not track this data for 2009-11 inclusive.

Based on everything we know, we believe this is almost certainly the first time the sector has raised over one billion dollars. 

Hope that helps.


Friday, January 4, 2013

2013 Biotech Showcase Cell Therapy and Regenerative Medicine Presentations #BTS13


While the Alliance of Regenerative Medicine has published a list of presentations by its members, this is not inclusive of all sector-related presentations so we've made our own list.  In case it is useful, here is our schedule of the 2013 EBD Biotech Showcase cell therapy and regenerative medicine presentations.

Downloadable versions: (Excel) (PDF)

Text version:

Monday, January 7, 2013
  Room Hearst - Track A
  10:00  Adaptimmune

  Mission Room II - Track C
  09:30  International Stem Cell

  Powell Room - Track D
  09:00  Cryoport

Tuesday, January 8, 2013
  Hearst Room - Track A
  15:00  TissueGene

  Mission I Room - Track B
  09:15  DC Prime
  10:00  MaxCyte
  10:15  Capricor
  10:30  Juventas Therapeutics
  11:00  PharmaCell
  11:15  DiscGenics
  11:30  SanBio
  11:45  Histogenics
  13:45  RhinoCyte

  Mission Room II – Track C
  10:00  Avita Medical 
  16:30  ViaCyte

  Powell Room - Track D
  10:00  Advanced Cell Technology
  10:30  ReNeuron Group
  11:00  Q Therapeutics
  11:30  Cytori Therapeutics
  13:45  Organovo
  14:15  Shire Regenerative Medicine
  14:45  Tigenix
  15:15  Athersys, Inc.
  15:45  Neuralstem
  16:15  Amorcyte

Wednesday, January 9, 2013
  Hearst Room - Track A
  10:30  TVAX Biomedical
  15:45  Aderans Research

  Mission Room I - Track B
  15:00  America Stem Cell
  15:15  Auxocell Laboratories

  Powell Room - Track D
  10:30  InVivo Therapeutics
  16:15  Medistem

I hope to see you there.


Tuesday, January 1, 2013

Cell Therapy Blog welcomes 2013


Happy new year to all our readers.  We look forward to our interactions throughout 2013. This month watch for:
We look forward to seeing you on the 2013 conference circuit.  For a complete and current list of 2013 cell therapy industry conferences, click here.

We will be in San Francisco next week during EBD Biotech Showcase and JP Morgan as well as at the Phacilitate Cell and Gene Therapy Forum in Washington, DC at the end of the month.

As always we welcome your comments, feedback, criticisms, and questions.

Thank you for all for everything to contributed to and did to support this blog and our efforts this past year.  Let's have a great 2013!

p.s.  Don't forget to follow Cell Therapy Blog on Twitter @celltherapyblog